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Review
. 2023:1430:71-89.
doi: 10.1007/978-3-031-34567-8_5.

United States Food and Drug Administration Regulation of Human Cells, Tissues, and Gene Therapies

Sandhya Sanduja  1 , Liz Lessey-Morillon  2 , Rondine Allen  2 , Xiaofei Wang  2 , Gavin Imperato  2 , Judith Arcidiacono  2
Affiliations
Review

United States Food and Drug Administration Regulation of Human Cells, Tissues, and Gene Therapies

Sandhya Sanduja et al. Adv Exp Med Biol. 2023.

Abstract

Research and development of gene therapies and cell- or tissue-based therapies has experienced exponential growth in recent decades and the potential for these products to treat diverse, often rare, clinical indications is promising. The Office of Therapeutic Products (OTP) in the Center for Biologics Evaluation and Research (CBER) at the United States Food and Drug Administration (US FDA) is responsible for the regulation of these products, among others, throughout the entire product lifecycle. This chapter provides an overview of the science- and data-driven approach to US FDA regulatory oversight of cell and gene therapy (CGT) products to ensure their safety and efficacy.

Keywords: Cell therapy; Clinical development; Clinical trial; Gene therapy; Investigational New Drug application; Marketing application; United States Food and Drug Administration.

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References

    1. Guidance for Industry: Long Term Follow-Up After Adminisatration of Human Gene Therapy Products, 2020. https://www.fda.gov/media/113768/download
    1. Guidance for Industry: Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), 2020. https://www.fda.gov/media/113790/download
    1. Guidance for Industry and Food and Drug Administration Staff: Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use, 2020. https://www.fda.gov/media/109176/download
    1. Cellular and Gene Therapy Guidances, 2022. https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/cellula... .
    1. Formal Meetings Between FDA and Sponsors or Applicants of PDUFA Products Guidance for Industry. December, 2017. https://www.fda.gov/media/109951/download

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